Advancing treatments for rare diseases

We are accelerating life-saving therapies tailored to rare disease patients in need.

Who Are We

Our company is dedicated to helping commercial, non-profit and academic partners achieve biopharmaceutical development and human health goals. By collaborating with world-renowned academics and clinicians, we help clients better understand complex disease-related issues and develop life-saving gene therapies for patients with rare and ultra-rare diseases that are overlooked and undertreated by traditional drug development efforts.

Highly
qualified
employees
Over
98% client
retention rate
Successfully
performed
multiple
projects
Years
of experience
in drug
development

How We Do It

Rare diseases are diverse and the total number of patients is extremely large. There are as many as 300 million rare disease patients worldwide. Addressing the treatment challenges of these patients is of great significance. Despite advances in science and technology, developing a new drug requires hundreds of millions of dollars and years of investment. This cost raises challenges for developing treatments for rare and ultra-rare disease populations.

Our collaborative framework reimagines the drug development process as technologies such as DNA recombination and gene cloning mature. We are committed to the development of treatments for a variety of rare diseases, primarily, providing an accelerated pathway to the development of life-saving therapeutics. We provide professional scientific services and technical support for industry collaborators to expand the orphan drug development pipeline.

Your Proven Partner for Rare Disease Development

Our company has been at the forefront of supporting new therapeutic development for many years. We are proud of our extensive and growing contribution to supporting rare disease therapy clients, especially. With our comprehensive end-to-end portfolio, we have comprehensive products and specialized services, along with scientific and regulatory experts, to fully facilitate your integrated project from start to finish.

Featured Services

Our company not only provides global customers with a wide range of services for rare disease genomic analysis, vector and strategy design, but also provides efficacy testing, safety and toxicology analysis for therapeutic products.

Featured Products

For many years, we have been at the forefront of supporting the development of new therapies for rare diseases. With our comprehensive end-to-end portfolio, we have a full range of products and services, along with scientific and regulatory experts to support you throughout your integrated program. You can contact us for a full list of products.

Adeno-associated Virus (AAV)
Adenovirus
Lentivirus
Plasmid
Animal Models

Develop Your Product with Support From Scientific Experts

Products require a high level of safety combined with a strong ability to infect cells to achieve therapeutic effects. The product's infectivity must be maintained throughout its shelf-life. Developing a stable drug product is challenging and requires experienced scientists, suitable analytical tools, and an adaptable development plan. Our company has extensive experience in successful formulation development and a strong scientific background in virus formulation, particle analysis, and infectivity assay development. Our experts are happy to help you develop for rare diseases and develop and characterize products.

Development Services for Product

We offer you a vast portfolio of development services for rare disease products. We offer a variety of services such as formulation development, lyophilization process development, stability studies, and more to meet your expectations and current development stage needs. The scientific experience and advice of our expert scientists will help you achieve your development goals faster and with a higher chance of success.

Analytical Services for Product

We offer standardized analytical methods for many common sample types, such as aggregation analysis, particle characterization, and particle identification. We select the method best suited for your drug discovery project from a broad range of analytical techniques in various categories and perform method development dedicated to your analytical challenges and drug product.